Alnylam announced that it plans to showcase its R&D progress and platform innovation, including updates on multiple near- and mid-stage potential transformative therapies that represent blockbuster opportunities as its pipeline rapidly expands across multiple therapeutic areas. Alnylam scientists will provide an update on the TRITON Phase 3 program for its next-generation transthyretin (TTR) silencer, nucresiran, which has the potential for greater than 95% knockdown with twice-annual dosing. Vutrisiran is currently under review for the treatment of ATTR amyloidosis with cardiomyopathy by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) target action date of March 23, 2025.
Alnylam scientists will also present an update on the new ALN-HTT02 program, which has a highly differentiated exon-1-targeting approach to lower huntingtin (HTT) for Huntington's disease, a progressive, fatal disorder. ALN-HTT02 is being developed in partnership with Regeneron. Alnylam scientists will share updates on several other programs that recently entered the clinic.
This includes filing Investigational New Drug (IND) applications for nine new Alnylam-led programs by the end of 2025, including two in new tissues, two in the central nervous system (CNS), and five in the liver. At R&D Day, Alnylam will present preclinical data that demonstrate the potential for a highly desirable product profile, including approximately 90% knockdown of the target 22 days after a low-dose, subcutaneous injection, and improved insulin sensitivity without weight gain. Alnylam Platform Innovation Will Drive the RNAi Revolution into the Future: Alnylam will strive to continue to provide best-in-class delivery solutions as it extends the applications for RNAi therapeutics.
Alnylam scientists will present new preclinical data on delivery solutions with best-in-class potential for adipose, muscle, heart, and kidney tissue. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding the potential for Alnylam to identify new potential drug development candidates and to advance its research and development programs, including statements regarding the number of INDs that Alnylam intends to file, the timing of initiation of any of Alnylam's clinical trials, and the achievement of pipeline milestones and data, including relating to ongoing or planned clinical trials of nucresiran, zilebesiran, mivelsrian, ALN-HTT02, ALN-6400 and other product candidates; Alnylam's ability to address additional diseases and to deliver RNAi therapeutics to all major tissue types by 2030 and to realize the full potential of its pioneering technology; Alnylam's ability To address additional diseases and to deliverRNAi therapeutics to all major tissues types by 2030 and to realize The full potential of its pioneering technology. Alnylam's ability to identify new potential drug development candidate and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally and will be available in partnership with Regeneron.
